RARE - Ultragenyx Pharmaceutical Inc. (RARE) Q4 2023 Earnings Call Transcript
2024-02-15 23:55:23 ET
Call End:
Ultragenyx Pharmaceutical, Inc. (RARE)
Q4 2023 Earnings Conference Call
February 15, 2024, 05:00 PM ET
Company Participants
Joshua Higa - Vice President of Investor Relations
Emil Kakkis - Chief Executive Officer & President
Erik Harris - Chief Commercial Officer
Howard Horn - Chief Financial Officer
Eric Crombez - Chief Medical Officer
Conference Call Participants
Gena Wang - Barclays
Maurice Raycroft - Jefferies
Anupam Rama - JPMorgan
Tazeen Ahmad - Bank of America
Dae Gon Ha - Stifel
Mehdi Goudarzi - Truist Securities
Brendan Smith - TD Cowen
Kristen Kluska - Cantor Fitzgerald
Lidia Rachkova - Goldman Sachs
Ashu Kumar - Citi
Jeffery Hung - Morgan Stanley
Will Raphael - Leerink Partners
Jack Allen - Baird
Thomas Hoffmann - H.C. Wainwright & Co., LLC
Presentation
Operator
Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2023 Financial Results Conference Call. . At this time, all participants are in a listen-only mode. At the end of the prepared remarks, you will have an opportunity to ask questions during the Q&A portion of the call. It is now my pleasure to turn to Josh -- turn the call to Joshua Higa, Vice President of Investor Relations.
Joshua Higa
Thank you. We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. Joining me on this call are Emil Kakkis, Chief Executive Officer and President, Erik Harris, Chief Commercial Officer; Howard Horn, Chief Financial Officer; and Eric Crombez, Chief Medical Officer. I'd like to remind everyone that during today's call, we will be making forward-looking statements. These statements are subject to certain risks and uncertainties and our actual results may differ materially. Please refer to the risk factors discussed in our latest SEC filings. I'll now turn the call over to Emil.
Emil Kakkis
Thanks, Josh, and good afternoon, everyone. In 2023, we generated significant momentum across our commercial and clinical programs that have set us up for Catalyst bridge 2024. On the commercial front, we progressed international regulatory and reimbursement negotiations across our marketed products, continuing to add to new approvals and positive reimbursement decisions. This geographic expansion along with growing demand in our existing markets put us in position to maintain our trajectory of robust year-over-year growth.
On the clinical side, we released new data on our key programs in 2023 and successfully advanced enrollment of our priority programs that can accelerate value creation for our company this year and in the coming years. At our Analyst Day in October, we shared exciting data from three programs that all will have additional data catalysts in 2024.
On UX 143 for osteogenesis imperfecta, the interim Phase II data showed substantial increases in bone real density and Z score after just six months of treatment. This led to a 67% reduction in analyzed fracture with 20 of the 24 patients showing no radiographically confirmed fractures at six months.
On GTX-102 for Angelman syndrome, data from the long-term extension cohorts Phase I/II study demonstrated clinically meaningful improvements across multiple neuro-developmental domains, including cognition, receptive communication, growth motors, behavior and fleet. These data show we can repeatedly dose GTX-102 for much longer than one year with acceptable safety profile....
Ultragenyx Pharmaceutical, Inc. (RARE) Q4 2023 Earnings Call Transcript